CA3145567A1 - Molecule d'acide nucleique oligomere activant le gene atoh1 et son utilisation - Google Patents
Molecule d'acide nucleique oligomere activant le gene atoh1 et son utilisation Download PDFInfo
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- 238000001542 size-exclusion chromatography Methods 0.000 description 1
- 239000011780 sodium chloride Substances 0.000 description 1
- 239000007787 solid Substances 0.000 description 1
- 229960005322 streptomycin Drugs 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 125000001424 substituent group Chemical group 0.000 description 1
- 230000000946 synaptic effect Effects 0.000 description 1
- 230000002194 synthesizing effect Effects 0.000 description 1
- YLQBMQCUIZJEEH-UHFFFAOYSA-N tetrahydrofuran Natural products C=1C=COC=1 YLQBMQCUIZJEEH-UHFFFAOYSA-N 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 229940104230 thymidine Drugs 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 230000002103 transcriptional effect Effects 0.000 description 1
- 230000008733 trauma Effects 0.000 description 1
- AVBGNFCMKJOFIN-UHFFFAOYSA-N triethylammonium acetate Chemical compound CC(O)=O.CCN(CC)CC AVBGNFCMKJOFIN-UHFFFAOYSA-N 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 210000004881 tumor cell Anatomy 0.000 description 1
- 230000005740 tumor formation Effects 0.000 description 1
- 241000701161 unidentified adenovirus Species 0.000 description 1
- 238000010200 validation analysis Methods 0.000 description 1
- 231100000889 vertigo Toxicity 0.000 description 1
- 230000001720 vestibular Effects 0.000 description 1
- 238000005406 washing Methods 0.000 description 1
- 238000001262 western blot Methods 0.000 description 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
- A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
- A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
- A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
- A61K31/708—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Abstract
Acide nucléique oligomère et son utilisation dans le traitement de la perte auditive. L'invention concerne également une petite molécule d'acide nucléique d'activation pour le traitement de la perte auditive. La petite molécule d'acide nucléique d'activation peut être une molécule d'ARN double brin ou simple brin ciblant la région de promoteur d'un gène ATOH1, comprenant un premier brin d'acide nucléique et un second brin d'acide nucléique. La molécule d'ARN double brin ciblant la région de promoteur du gène ATOH1 comprend deux brins d'acide nucléique de 16 à 35 nucléotides de longueur, et l'un ou l'autre des brins d'acide nucléique présente au moins 75 % d'homologie ou de complémentarité avec une cible choisie dans la région promotrice du gène ATOH1. L'utilisation de la petite molécule d'acide nucléique d'activation et l'utilisation de la composition pharmaceutique contenant éventuellement un vecteur pharmaceutiquement acceptable peuvent augmenter l'expression du gène ATOH1 et de la protéine dans des cellules et traiter des maladies ou des affections liés à une expression insuffisante ou réduite du gène ATOH1.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN201910598221 | 2019-07-04 | ||
| CN201910598221.6 | 2019-07-04 | ||
| PCT/CN2020/100093 WO2021000928A1 (fr) | 2019-07-04 | 2020-07-03 | Molécule d'acide nucléique oligomère activant le gène atoh1 et son utilisation |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3145567A1 true CA3145567A1 (fr) | 2021-01-07 |
Family
ID=74100241
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3145567A Pending CA3145567A1 (fr) | 2019-07-04 | 2020-07-03 | Molecule d'acide nucleique oligomere activant le gene atoh1 et son utilisation |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220313721A1 (fr) |
| EP (1) | EP3995578A4 (fr) |
| JP (1) | JP2022539770A (fr) |
| KR (1) | KR20220029656A (fr) |
| CN (1) | CN114174511A (fr) |
| AU (1) | AU2020298647A1 (fr) |
| CA (1) | CA3145567A1 (fr) |
| WO (1) | WO2021000928A1 (fr) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111849968A (zh) | 2019-04-30 | 2020-10-30 | 中美瑞康核酸技术(南通)研究院有限公司 | 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用 |
| CN117448381B (zh) * | 2023-10-26 | 2024-06-21 | 上海交通大学医学院附属第九人民医院 | 基因编辑激活Atoh1转录促进前庭毛细胞再生及修复前庭功能 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8188131B2 (en) * | 2008-02-07 | 2012-05-29 | Massachusetts Eye & Ear Infirmary | Compounds that enhance Atoh1 expression |
| US8895528B2 (en) * | 2010-05-26 | 2014-11-25 | Curna, Inc. | Treatment of atonal homolog 1 (ATOH1) related diseases by inhibition of natural antisense transcript to ATOH1 |
| CN104630219B (zh) * | 2013-11-15 | 2019-05-07 | 中美瑞康核酸技术(南通)研究院有限公司 | 抑癌基因IRX1的saRNA分子及其组合物和其应用 |
| WO2015105191A1 (fr) * | 2014-01-10 | 2015-07-16 | 学校法人順天堂 | Méthode d'évaluation des lésions lymphadénopathiques |
| KR20230145206A (ko) * | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| CN106032532A (zh) * | 2015-03-17 | 2016-10-19 | 中国医学科学院北京协和医院 | 一种小激活rna及其制备方法和应用 |
| CN106480028B (zh) * | 2016-10-12 | 2019-04-05 | 上海市第七人民医院 | TPO基因的saRNA分子及其应用 |
-
2020
- 2020-07-03 CA CA3145567A patent/CA3145567A1/fr active Pending
- 2020-07-03 WO PCT/CN2020/100093 patent/WO2021000928A1/fr not_active Ceased
- 2020-07-03 CN CN202080045801.3A patent/CN114174511A/zh active Pending
- 2020-07-03 AU AU2020298647A patent/AU2020298647A1/en not_active Abandoned
- 2020-07-03 JP JP2021577888A patent/JP2022539770A/ja active Pending
- 2020-07-03 EP EP20834537.1A patent/EP3995578A4/fr not_active Withdrawn
- 2020-07-03 KR KR1020227000881A patent/KR20220029656A/ko active Pending
- 2020-07-03 US US17/597,281 patent/US20220313721A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| KR20220029656A (ko) | 2022-03-08 |
| AU2020298647A1 (en) | 2022-02-24 |
| EP3995578A4 (fr) | 2023-07-26 |
| WO2021000928A1 (fr) | 2021-01-07 |
| US20220313721A1 (en) | 2022-10-06 |
| CN114174511A (zh) | 2022-03-11 |
| EP3995578A1 (fr) | 2022-05-11 |
| JP2022539770A (ja) | 2022-09-13 |
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